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Trial registered on ANZCTR


Registration number
ACTRN12612000652875
Ethics application status
Approved
Date submitted
7/06/2012
Date registered
19/06/2012
Date last updated
1/12/2015
Type of registration
Prospectively registered

Titles & IDs
Public title
Study of the Drug BTD-001 in Young Adults and Adolescents with Down Syndrome
Scientific title
A Phase IB, Double-blind, Randomized, Placebo-Controlled, Parallel Group Study of the Safety, Tolerability, Preliminary Efficacy and Pharmacodynamics of BTD-001 in Young Adults and Adolescents with Down Syndrome
Secondary ID [1] 280642 0
BTD-001 DS102
Universal Trial Number (UTN)
Trial acronym
The COMPOSE Study
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Down syndrome 286657 0
Condition category
Condition code
Human Genetics and Inherited Disorders 286951 286951 0 0
Down's syndrome

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Participants (13 to 35 yrs in age) will be enrolled. Following screening, a visit prior to first dose will establish baseline measurements of cognitive function, adaptive behaviour scoring and evoked response measurements. Eligible participants will be randomised to one of the following treatments: Treatment A - 50mg BTD-001 taken orally twice daily, as oral solution. Treatment B - 100mg BTD-001 taken orally twice daily, as oral solution. Treatment C - Placebo taken orally twice daily, as oral solution All study treatments will be made up as a flavoured drink solution. Study drug will be given for 12 weeks.
Intervention code [1] 285039 0
Treatment: Drugs
Comparator / control treatment
Sterile purified water will be used as placebo made up as a flavoured drink and taken as oral solution.
Control group
Placebo

Outcomes
Primary outcome [1] 287295 0
Safety and tolerability of BTD-001 twice daily oral as assessed by: physical and neurological examinations; laboratory safety tests; ECG; vital signs; adverse event collection. Adverse events are any untoward medical occurrences in study participants administered study treatment.
Based on animal studies and the experience of other people who have received the active ingredient in BTD-001, side effects that may occur include feelings of anxiety and difficulty with sleeping. At very high doses, seizures (fits) and gastrointestinal effects (nausea and vomiting) have been reported to occur. Please note that these side effects have occurred at doses 10-20 times greater than those being tested in this study.
Timepoint [1] 287295 0
At 2 weeks, 4 weeks, 8 weeks, 12 weeks and 16 weeks
Secondary outcome [1] 297850 0
Cognitive, and behavioural/functional effects of BTD-001 in persons with Down syndrome as assessed by psychometric and functional measures. Psychometric measures include standardise tests assessments for functional and adaptative behavior and language. These include: Vineland adaptive behavior scale (VABS-II), Behavior rating inventory of executive function (BRIEF), and Clinical Evaluation of Language Functions (CELF4). Cognitive test assessments will be performed using a computer based testing system [CANTAB(Registered Trademark)].
Timepoint [1] 297850 0
At 4 weeks, 12 weeks, 16 weeks
Secondary outcome [2] 297851 0
Effects of BTD-001 on auditory event related potentials (ERP) in persons with Down syndrome. Auditory ERP will be assessed by electroencephalography (EEG).
Timepoint [2] 297851 0
At 4 weeks, 12 weeks, 16 weeks

Eligibility
Key inclusion criteria
1. Males and females, age 13–35 years with Down syndrome 2. Body weight greater than 40 kg AND BMI less than 40.0 kg/m2 3. Ability to cooperate and complete required tests and procedures. 4. Capable of undergoing the cognitive test battery and evoked response potential testing. 5. Negative pregnancy test AND non-lactating (females only) 6. If sexually active and female, the person with DS must be surgically sterile OR using reliable contraception 7. Laboratory findings, vital signs and ECG within normal limits or any abnormalities judged clinically not significant 8. Stable medical condition For Parents/Guardians 1. Parents or guardians of the person with DS must understand the study, give written and dated informed consent/assent, and successfully complete behavioural assessment interviews. 2. Parent or guardian agree to accompany the subject to all visits, be capable of supervising and reporting study drug use and adequately reporting the subject’s functional status.
Minimum age
13 Years
Maximum age
35 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Current use of anti-epileptics, focal seizure disorder, syncope, unexplained loss of consciousness at any time OR seizure in the past 3 years.
2. History of or concurrent neurological disorders interfering with cognition.
3. History or diagnosis of clinically significant psychiatric or neurological disease.
4. Neuroleptic agent, bilobalide-containing product OR benzodiazepine use in last 4 weeks
5. Significant endocrine abnormality
6. Subjects with hypothyroidism are excluded unless they have received a stable dose of medication.
7. Significant hepatic impairment
8. Significant renal impairment
9. History of porphyria
10. History of leukemia or other malignancy
11. Participation in a clinical drug trial within 4 weeks
12. Severe sleep apnea or excess daytime sleepiness
13. Recent or anticipated use of prohibited medications
14. Have any other condition which, in the Investigator’s opinion, would put the person with Down syndrome at risk by participating in this study

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Patients and their carers who consent to take part in the study will be allocated a unique subject number. After screening, eligible patients who meet the inclusion and exclusion criteria will be randomised to 1 of 3 blinded treatments,

An automated system will administer the randomization process and provide the Investigator codes used to assign study drug packaging to subjects.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
An automated system will administer the randomization process and provide the Investigator codes used to assign study drug packaging to subjects.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint(s)
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,TAS,WA,VIC
Recruitment postcode(s) [1] 9791 0
2031 - Randwick
Recruitment postcode(s) [2] 9793 0
2050 - Camperdown
Recruitment postcode(s) [3] 9794 0
2050 - Camperdown
Recruitment postcode(s) [4] 9796 0
2522 - University Of Wollongong
Recruitment postcode(s) [5] 9801 0
3122 - Hawthorn
Recruitment postcode(s) [6] 7546 0
3168 - Notting Hill
Recruitment postcode(s) [7] 9802 0
4101 - South Brisbane
Recruitment postcode(s) [8] 9799 0
4215 - Southport
Recruitment postcode(s) [9] 9792 0
5000 - Adelaide
Recruitment postcode(s) [10] 9800 0
5034 - Clarence Park
Recruitment postcode(s) [11] 9795 0
6008 - Subiaco
Recruitment postcode(s) [12] 9797 0
6009 - Broadway Nedlands
Recruitment postcode(s) [13] 9798 0
7250 - Launceston
Recruitment outside Australia
Country [1] 4352 0
New Zealand
State/province [1] 4352 0

Funding & Sponsors
Funding source category [1] 285409 0
Commercial sector/Industry
Name [1] 285409 0
Balance Therapeutics, Pty Ltd.
Address [1] 285409 0
Balance Therapeutics, Pty Ltd 2/1518 Nepean Highway, Mount Eliza VIC 3930 Australia
Country [1] 285409 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Novotech (Australia) Pty Limited
Address
Level 3, 235 Pyrmont Street
Pyrmont NSW 2009
Country
Australia
Secondary sponsor category [1] 284261 0
None
Name [1] 284261 0
Address [1] 284261 0
Country [1] 284261 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 287421 0
Monash University Human Research Ethics Committee
Ethics committee address [1] 287421 0
1st floor, Building 3e, Monash Research Office, Clayton Campus, Monash University, Wellington Road, Clayton, VIC 3800
Ethics committee country [1] 287421 0
Australia
Date submitted for ethics approval [1] 287421 0
Approval date [1] 287421 0
12/03/2012
Ethics approval number [1] 287421 0
CF11/3234 - 2011001763

Summary
Brief summary
BTD-001, has been used for several decades around the world as a treatment for dementia, a respiratory stimulant and as an ingredient of a cough medication. Studies in a mouse model of Down syndrome (DS) (Ts65Dn transgenic mouse) have demonstrated that chronic administration of the product improved cognition on a number of behavioural assessments. These improvements were sustained months after discontinuation of drug administration, indicating that BTD-001 may cause long-lasting synaptic changes supporting improved cognition. These data indicate that BTD-001, may improve function and cognition in persons with DS and has the potential to improve educational and quality of life outcomes for this population.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 34287 0
Prof Rhoshel Lenroot
Address 34287 0
Neuroscience Research Australia
Barker Street
Randwick, NSW 2031
Country 34287 0
Australia
Phone 34287 0
+61 2 9399 1089
Fax 34287 0
Email 34287 0
r.lenroot@unsw.edu.au
Contact person for public queries
Name 17534 0
Mrs Sasita Sophia
Address 17534 0
Novotech (Australia) Pty Ltd
Level 3, 235 Pyrmont St
Pyrmont, NSW 2009
Country 17534 0
Australia
Phone 17534 0
+61 2 8569 1400
Fax 17534 0
+61 2 8569 1498
Email 17534 0
Sasita.sophia@novotech-cro.com
Contact person for scientific queries
Name 8462 0
Prof Rhoshel Lenroot
Address 8462 0
Neuroscience Research Australia
Barker Street
Randwick, NSW 2031
Country 8462 0
Australia
Phone 8462 0
+61 2 9399 1089
Fax 8462 0
Email 8462 0
r.lenroot@unsw.edu.au

No information has been provided regarding IPD availability
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary